Parents await decision on groundbreaking drug for their children

FAMILIES are hoping a groundbreaking drug which helps children with a rare condition to walk will be approved for use on the NHS in Scotland today.

The treatment, Translarna, is the first treatment ever to address the underlying cause of Duchenne muscular dystrophy and it helps young sufferers – who often depend on wheelchairs by the age of eight – to stay on their feet longer. While the drug has been available to patients in other European countries such as France, Spain and Germany for more than a year, families in the UK have been waiting 18 months to hear if the NHS will prescribe the treatment.

Now the Scottish Medicines Consortium (SMC), which evaluates new drugs to decide if they should be available on the NHS, is due to announce its decision.

Michelle Young, whose son Michael suffers from Duchenne muscular dystrophy, said: “It’s been a long wait for us as a family to find out the SMC’s answer on Translarna.”

Earlier this year, nine-year-old Michael, from Larbert, wrote a letter to First Minister Nicola Stur- geon appealing for her help to ensure patients like himself could have the treatment. He had been given the drug as part of a trial but was worried what would happen as the trial had finished. He told Ms Sturgeon: “If my medicine is stopped I will need a wheelchair.”

Ms Young described Translarna as the first piece of a jigsaw puzzle in treating his condition: “It is putting off some very difficult challenges for Michael, but, also, it is buying time.The cost to the NHS of ignoring the reality of life with Duchenne is great.”

Cormac Fegan, aged four from Longniddry in East Lothian, is the only child in the UK to receive Translarna on the NHS after a special request for funding was made.

His father, Gary Fegan, said: “It’s my greatest hope that the SMC will approve Translarna for Cormac and other children like him. Whilst Cormac has only been on Translarna for five months, the difference we’ve seen in him, not only physically, but also in terms of his behaviour, has been incredible. There’s nothing to suggest that this won’t continue.”